Challenges in prophylactic therapy with Emicizumab in patients with hemophilia A: Focus on monitoring tests

Abstract

Abstract This study presents a transversal investigation that we performed at Fundeni hospital (Bucharest, Romania) into the therapeutic benefits and efficacy of Emicizumab, a non-factor therapy, in the context of hemophilia A. Ten patients diagnosed with hemophilia A were closely monitored using clinical and laboratory resources during Emicizumab treatment, with an average of 12.8 months. Among these patients, six exhibited anti-factor VIII inhibitors, changing the medical strategy and adding complexity to their clinical profiles. A comprehensive approach was adopted to assess the coagulation status of patients under Emicizumab therapy. The study employed several key coagulation monitoring tools, including thrombin generation time (TGT) and thrombelastography (TEG). These methodologies generated valuable results in evaluating the patients’ coagulation profiles during the treatment regimen. Additionally, traditional coagulation assays were utilized to gain a profound understanding of the overall coagulation dynamics and to evaluate the therapeutic response. During prophylaxis with Emicizumab all patients experienced a reduced number of bleeding events. Moreover, a subset of these patients underwent major surgical procedures (orthopedic joint replacements, cholecystectomy) with successful outcomes. These findings underscore the potential of Emicizumab therapy as an effective option for hemophilia A patients, including those with inhibitors. Our research provides physicians several insights, offering a potential avenue for improved patients’ care and treatment strategies that translate in enhanced quality of life for hemophilia A patients undergoing Emicizumab therapy.